The Newman-Lakka Institute for Personalized Cancer Care at Tufts Medical Center in Boston will employ innovative statistical tools to study the effectiveness in children of personalized cancer treatments approved for use in adults.

Analytical tools compare observed and predicted outcomes for children with cancer.

The Newman-Lakka Institute for Personalized Cancer Care at Floating Hospital for Children at Tufts Medical Center in Boston, established in July under a $2.5 million private grant, aims to study the effectiveness in children of personalized cancer treatments already approved for use in adults.

To do this, the Institute is using a new biostatistical approach to clinical research.

With the shift toward individualized treatments that target specific molecular pathways in cancer, it grows much more difficult to gather enough children receiving the same treatment to study in a traditional clinical trial. The key to speeding this process, says Johannes Wolff, MD, clinical director of the Newman-Lakka Institute, “is to be able to work without a traditional control group.”

Instead of the traditional approach of comparing an experimental group to a control group, Wolff has developed a novel database and innovative biostatistical analysis tools that allow him and his colleagues to compare a group of patients receiving personalized treatment to all other patients in the database regardless of their treatment. More specifically, Wolff's database compares the differences between a statistically predicted outcome and an observed outcome. “That is the key that allows us to tell how well a treatment worked even for individualized medicine,” says Wolff.

“To put it simply, we use a database containing historical and concurrent controls with the same disease,” says Giannoula Lakka Klement, MD, scientific director of the Newman-Lakka Institute.

In July in Pediatric Blood Cancer, Wolff and his co-researchers published his first successful analysis using the tools for pediatric brain tumors. They showed outcome benefits for a small group of heterogeneous patients treated with personalized targeted therapies compared with a large collection of prospective data from patients treated differently.

The Newman-Lakka Institute is also developing novel biomarkers to assess how a tumor is responding to treatment. Markers beyond tumor size, such as measures of angiogenesis regulators in platelets, will give clinicians additional ways to assess whether the targeted treatment is worth continuing or whether it should be changed, says Klement.

In addition to the Tufts Medical Center Institute, 4 centers are enrolling patients for the project: the Masonic Cancer Center at the University of Minnesota in Minneapolis, Miami Children's Hospital, the New York University Cancer Institute, and The University of Texas MD Anderson Cancer Center in Houston. Once the kinks in the system are worked out, says Wolff, “sharing it with additional centers is the next step.”